Jan 8, 2020

Sangamo Therapeutics has handed over development of hemophilia A gene therapy candidate SB-525 to Pfizer. Up to this point, the investigational therapy had been developed by Sangamo, in collaboration with Pfizer.

SB-525, which is now advancing to phase 3 clinical trials, is developed using recombinant adeno-associated viruses (AAVs) as vectors to deliver the genetic codes that illicit factor VIII production in hemophilia A patients. These AAVs deliver the modified genetic material into an individual’s liver cells without causing disease or triggering significant immune responses.

This most recent announcement follows last month’s American Society of Hematology Annual Meeting, where the two companies announced positive updated results from the phase 1/2 Alta study designed to assess both the safety and tolerability of SB-525 in patients with severe hemophilia A.

According to a recent press release Pfizer has already begun patient recruitment for the phase 3 “lead-in trial,” a six-month study designed to evaluate the current effectiveness and safety of preventive replacement therapy in the usual care setting. The lead-in study will support SB-525’s advancement to phase 3 registrational clinical trials.

“I want to congratulate our team for their success in developing SB-525 through to this important milestone where we have handed over the IND to Pfizer for phase 3 development,” said Sandy Macrae, CEO of Sangamo.

“We are thrilled to be in a partnership where both parties have cooperated to accelerate study timelines, resulting in completion of the IND transfer ahead of schedule. Pfizer and Sangamo are united in our common interest to help patients with hemophilia A and will do everything that we can to safely and expeditiously advance this promising gene therapy candidate for patients in need,” added Macrae.

Source: Hemophilia News Today, January 6, 2020